Rare diseases

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Disc maps out US filing for porphyria drug bitopertin

Disc Medicine has charted a route to market for bitopertin, its drug candidate for rare disease erythropoietic protoporphyria (EPP), after meeting with the FDA.

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Biogen shows its IgAN hand with felzartamab data

Biogen has revealed encouraging phase 2 results with its anti-CD38 antibody felzartamab in rare kidney disease IgA nephropathy (IgAN), which it hopes could join a series of first-in-class t

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kidney disease computer graphic scan image

Novartis eyes year-end filing in US for Fabhalta in C3G

Novartis is cueing up a new indication in the US for its oral therapy for complement-mediated diseases – Fabhalta – after reporting new data with the drug in ultra-rare kidney disease C3 gl

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AZ, Ionis get CHMP backing for ATTR polyneuropathy drug

AstraZeneca and Ionis are set fair for EU approval of Wainzua, their treatment for polyneuropathy associated with transthyretin-mediated amyloidosis (ATTR), after the EMA's human medicines

News

Lundbeck drops in on epilepsy drug with $2.6bn Longboard bid

Neuroscience specialist Lundbeck has agreed to buy Longboard Pharmaceuticals for $2.6 billion, expanding its pipeline with an epilepsy drug tipped as a future blockbuster.

News

FDA knocks back Zealand's dasiglucagon once again

Zealand Pharma has suffered another setback in its bid to bring glucagon receptor agonist dasiglucagon for ultra-rare disease congenital hyperinsulinism (CHI) to the US market.

Disc maps out US filing for porphyria drug bitopertin

Biogen shows its IgAN hand with felzartamab data

Novartis eyes year-end filing in US for Fabhalta in C3G

AZ, Ionis get CHMP backing for ATTR polyneuropathy drug

Lundbeck drops in on epilepsy drug with $2.6bn Longboard bid

FDA knocks back Zealand's dasiglucagon once again

FDA experts lukewarm on AZ's anticoagulant antidote Andexxa

Coming soon: The Life Sciences Industry Report 2025 – insigh...

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